A new UCL spin-out company – Autolus – is being launched today to develop and commercialise a new generation of engineered T-cell therapies for haematological and solid tumours, with the backing of £30m in investment from healthcare investment company Syncona.
UCLB patented technology shows recovery of vision in glaucomatus rats
8 March 2012
Scientists funded by the Medical Research Council have successfully used adult human retinal stem cells to repair nerve cells damaged in glaucoma, partially restoring vision in rats.
If the success can be replicated in humans it may eventually lead to new treatments for glaucoma, the leading cause of irreversible blindness in the world, and other degenerative eye conditions by slowing sight deterioration, or reversing it altogether. The research is published in the journal Stem Cells Translational Medicine.
Glaucoma occurs when a build up of pressure in the eye leads to the death of retinal ganglion cells (RGCs), which form the nerve fibres of the optic nerve and that transmit visual information from the eye to the brain. The condition can be treated in the early stages, but if diagnosis is late or treatment doesn't work, damage to the eye cannot be reversed.
The researchers looked at whether injecting a type of adult human stem cell, known as the Müller glia stem cell, could stimulate repair of damaged RGCs in an animal model. Müller glia are found in the retina of humans and other vertebrates and are multipotent, meaning they can grow into any of the different nerve cells found in the retina.
The researchers used chemicals to induce Müller glia to grow into precursors of RGCs, before transplanting them onto the retinas of rats which had damage to their own RGCs.
After four weeks, the injected cells appeared to have formed new connections (synapses) with existing nerve cells and the rats had significantly improved retinal function when their vision was tested under very low light conditions. The scientists hope that, with further work, they will be able to develop new treatments to improve or restore vision in patients with untreatable glaucoma.
Dr Astrid Limb, who led the study at the UCL Institute of Ophthalmology, UCL, said:
"Although this research is still a long way from the clinic, it is a significant step towards our ultimate goal of finding a cure for glaucoma and other related conditions. We are optimistic that after further work on animal models to perfect our transplantation technique we will be in a good position to start early-stage clinical trials on humans in around three to five years.
"The human eye is actually very efficient. We can still have fairly good vision with very few functioning retinal nerve cells, which is why many glaucoma patients don't show symptoms until it is too late to treat the underlying cause of their vision loss. If we can restore even a small number of RGCs through cell therapy, and achieve functioning vision, we could potentially delay or even reverse blindness caused by glaucoma."
Professor Peng T Khaw, director of the NIHR Biomedical Research Centre at Moorfields and the Institute of Ophthalmology and a co-author of the paper, said:
"These results are very exciting. We see patients with glaucoma whose lives would be transformed with an improvement of only a small percentage of nerve cell function. The results of these experiments suggest that this may be possible in the future using cells we all have in our own eyes to achieve this. Translational research like this gives hope to the many millions of people who have lost vision due to glaucoma."
Dr Rob Buckle, head of regenerative medicine at the MRC, said:
"Regenerative medicine is a key priority for the MRC and it's wonderful to see another example of how our significant investment in stem cell research in recent years is beginning to deliver results. Repair of the eye is an area that is now at the forefront of this field, and this study highlights a new route for delivering the promise of regenerative medicine to treat disabling conditions such as glaucoma."
Dr Rachel Hemsley, Senior Business Manager at UCLB said:
"Glaucoma is the second largest cause of blindness in the world with an estimated 65 million people suffering from it. The potential to create a therapy and recover vision for patients is very exciting. We are working with the team to translate key research findings into a product which addresses a clear, unmet medical need both within the UK and worldwide."
The research was carried out by scientists at the UCL Institute of Ophthalmology and Moorfields Eye Hospital, London. It was funded by the MRC, the Helen Hamlyn Trust, Fight for Sight, Moorfields Special Trustees, and the NIHR BRC at Moorfields Eye Hospital and the UCL Institute of Ophthalmology. Patents to this technology are held by UCLB.
About the Medical research Council
For almost 100 years the Medical Research Council has improved the health of people in the UK and around the world by supporting the highest quality science. The MRC invests in world-class scientists. It has produced 29 Nobel Prize winners and sustains a flourishing environment for internationally recognised research. The MRC focuses on making an impact and provides the financial muscle and scientific expertise behind medical breakthroughs, including one of the first antibiotics penicillin, the structure of DNA and the lethal link between smoking and cancer. Today MRC funded scientists tackle research into the major health challenges of the 21st century. www.mrc.ac.uk
About Moorfields Eye Hospital/UCL Institute of Ophthalmology
Moorfields Eye Hospital/UCL Institute of Ophthalmology NIHR Biomedical
Research Centre is a partnership between Moorfields Eye Hospital NHS
Foundation Trust and the UCL Institute of Ophthalmology. Established
in April 2007, its purpose is to conduct 'translational research' that is
designed to take advances in basic medical research from the laboratory to
the clinic, enabling patients to benefit more quickly from new scientific
breakthroughs. Our centre is currently one of 12 biomedical research
centres that were awarded in 2007 to NHS/university partnerships with an
outstanding international reputation for medical research and expertise,
and experience of translating that research into the clinical
setting. For further information, please visit www.brcophthalmology.org
UCLB is a leading technology transfer company that supports and
commercialises research and innovations arising from UCL, one of the
UK's top research-led universities. UCLB has a successful track record
and a strong reputation for identifying and protecting promising new
technologies and innovations from UCL academics. It invests directly in
development projects to maximise the potential of the research and
manages the commercialisation process of technologies from the
laboratory to market. UCLB supports UCL's Grand Challenges of increasing
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